FDA approves $2M medicine, most expensive ever

FDA approves $2M medicine, most expensive ever
This photo provided by Novartis shows Zolgensma. The one-time gene therapy developed by Novartis, Zolgensma, will cost $2.125 million. It treats a rare condition called spinal muscular atrophy, or SMA, which strikes about 400 babies born in the U.S. each year. The therapy, given in a one-hour infusion, was approved for children under age 2 and will be available within two weeks. (Novartis via AP)

U.S. regulators have approved the most expensive medicine ever, for a rare disorder that destroys a baby's muscle control and kills nearly all of those with the most common type of the disease within a couple of years.

The is priced at $2.125 million. Out-of-pocket costs for patients will vary based on insurance coverage.

The medicine, sold by the Swiss drugmaker Novartis, is a gene therapy that treats an inherited condition called . The treatment targets a defective gene that weakens a child's muscles so dramatically that they become unable to move, and eventually unable to swallow or breathe. It strikes about 400 babies born in the U.S. each year.

The Food and Drug Administration on Friday approved the treatment, called Zolgensma, for all children under age 2 who are confirmed by a genetic test to have any of the four types of the disease. The therapy is a one-time infusion that takes about an hour.

Novartis said it will let insurers make payments over five years, at $425,000 per year, and will give partial rebates if the treatment doesn't work.

The one other medicine for the disease approved in the U.S. is a drug called Spinraza. Instead of a one-time treatment, it must be given every four months. Biogen, Spinraza's maker, charges a list price of $750,000 for the first year and then $350,000 per year after that.

The independent nonprofit group Institute for Clinical and Economic Review, which rates the value of expensive new medicines, calculated that the price of the new is justifiable at a cost of $1.2 million to $2.1 million because it "dramatically transforms the lives of families affected by this devastating disease."

ICER's president, Dr. Steven D. Pearson, called the treatment's price "a positive outcome for patients and the entire health system."

The that causes spinal muscular atrophy prevents the body from making enough of a protein that allows nerves that control movement to work normally. The nerves die off without the protein.

FDA approves $2M medicine, most expensive ever
This Aug. 13, 2016, file photo shows a logo of Swiss pharmaceutical company Novartis in Seoul, South Korea. On Friday, May 24, 2019, U.S. regulators approved Zolgensma, the most expensive medicine ever, a therapy meant to cure a disorder that rapidly destroys a baby's muscle control and kills most within a couple years. (AP Photo/Ahn Young-joon, File)

In the most common type, which is also the most severe, at least 90% of patients die by age 2, and any still alive need a ventilator to breathe. Children with less-severe types become disabled more slowly and can live for up to a couple decades.

Zolgensma works by supplying a healthy copy of the faulty gene, which allows to then start producing the needed protein. That halts deterioration of the nerve cells and allows the baby to develop more normally.

In patient testing, babies with the most severe form of the disease who got Zolgensma within 6 months of birth had limited muscle problems. Those who got the treatment earliest did best.

Babies given Zolgensma after six months stopped losing muscle control, but the medicine can't reverse damage already done.

It is too early to know how long the benefit of the treatment lasts, but doctors' hopes are rising that they could last a lifetime, according to Dr. Jerry Mendell, a neurologist at Nationwide Children's in Columbus, Ohio, who led one of the early patient studies.

"It's beginning to look that way," he said, because a few children treated who are now 4 or 5 still have no symptoms.

Early diagnosis is crucial, so Novartis has been working with states to get genetic testing for newborns required at birth. It expects most states will have that requirement by next year.

The FDA said side effects included vomiting and potential liver damage, so must be monitored for the first few months after treatment.


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User comments

May 24, 2019
How much does the medicine cost to manufacturer? Is it very expensive to make or are they charging that much only because of what the results can be?

May 25, 2019
Haven't seen anything so far beyond "because we can".

May 25, 2019
Included in the price of medicine is the cost of research into developing a medicine. This has to be recouped before the time that generic versions are allowed to be released. They also factor in profit to the cost, and the need for the medicine versus the alternative actions available.

It is priced very high though. It wouldn't take many doses to recover the cost of development and make a healthy profit. As carbon_unit mentioned, a large factor in this will be 'because we can'. The unfortunate thing is the majority will miss out, and those where the medicine is funded through health care or other schemes may mean others may miss out of a certain medicine due to funding distribution.

May 25, 2019
How much does the medicine cost to manufacturer? Is it very expensive to make or are they charging that much only because of what the results can be?


Who cares? Charge what the highest price that will maximize profits.

That's capitalism baby.

May 25, 2019
Included in the price of medicine is the cost of research into developing a medicine.


Is that why the cost of insulin - patent sold for a dollar - has increased by a 500 percent over the last 8 years?


May 25, 2019
Sure it's nice, but is it rational? How many lives can we save with $2 million? Is it a sign of compassion or demagogy? Why not trying to detect it at the featus stage so the mother could abort? Yes abortion isn't the best solution, but in that case it saves (or sacrifice) as many lives you could save for that price.

May 26, 2019
Abortion? Isn't that what Republicans call "baby murder".

I was just watching a video linked one conservative web site in which "proof" is provided that planned parenthood is "murdering children" and pumping their blood to the factories where fake meat is being produced under FDA approval and sold to fast food restaurants as a "green alternative" to real meat.

They even use Google Earth to show the Planned Parenthood office is right across the road from the fake meat factory.

-Q-


May 26, 2019
"Novartis said it will let insurers make payments over five years, at $425,000 per year, and will give partial rebates if the treatment doesn't work."

-So lessee, $2M a baby @ 400 babies a year, that's $800M, and you can pay over 5 years, but each year there are 400 more babies... I'm not good at math but this sure looks like compound interest to me.

What's an abortion cost nowadays?

"Nationwide, the cost at health centers ranges from about $350 to $950 for abortion in the first trimester. The cost is usually more for a second-trimester abortion. Costs vary depending on how long you've been pregnant and where you go."

-So hard to assign relative value isnt it?

May 27, 2019
There are only 780 abortion clinics in the entire U.S.

1 clinic for every half million people.

]\q]"Nationwide, the cost at health centers ranges from about $350 to $950 for abortion in the first trimester. The cost is usually more for a second-trimester abortion. Costs vary depending on how long you've been pregnant and where you go."

-So hard to assign relative value isnt it?

May 30, 2019
Put another way, we have kids hanging over a cliff with one hand, we could pull them up, but we let them fall instead.

By the way, most R&D costs were probably covered by public research.

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